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We've funded more than 2700 projects and 1600 researchers to give people affected by a rare genetic condition a new hope.
Our methodHow we fund research?
- 592.5 Millions of euros invested
- 1644 Researchers
- 2720 Projects funded
What we do
We were made to give people affected by a rare genetic condition an answer – treatment for their condition. Rare genetic conditions are not statistically relevant when considered individually, but the same can’t be said about a person’s life.
We support the best projects chosen using a rigorous and transparent selection process, following consultation with an internationally-renowned committee of qualified scientists.
Generating blood in vitro: a new piece of the puzzle
An important contribution to the elucidation of the mechanisms through which blood stem cells arise during embryonic life from a study conducted by the San Raffaele Telethon Institute for Gene Therapy.
Gene therapy targeted to the brain: a complex but not impossible challenge
Angela Gritti from the San Raffaele Telethon Institute of Milan talks about the efforts of applying gene therapy to one of the most difficult body organs to access.
Aicardi-Goutières syndrome: when the immune system detects viruses where there are none
An interview with Anna Kajaste-Rudnitski from the San Raffaele Telethon Institute for Gene Therapy in Milan, author of a study that gives new insights into this rare syndrome and provides also useful information to investigate the interaction between the immune system and viruses, including Sars-CoV-2.